Revertant mosaicism in skin: Natural gene therapy

Joey E. Lai-Cheong, John A. McGrath, Jouni Uitto

研究成果: Review article同行評審

103 引文 斯高帕斯(Scopus)

摘要

Revertant mosaicism is a naturally occurring phenomenon involving spontaneous correction of a pathogenic mutation in a somatic cell. Recent studies suggest that it is not a rare event and that it could be clinically relevant to phenotypic expression and patient treatment. Indeed, revertant cell therapy represents a potential 'natural gene therapy' because in vivo reversion obviates the need for further genetic correction. Revertant mosaicism has been observed in several inherited conditions, including epidermolysis bullosa, a heterogeneous group of blistering skin disorders. These diseases provide a useful model for studying revertant mosaicism because of the visual and accessible nature of skin. This overview highlights the latest developments in revertant mosaicism and the translational implications germane to heritable skin disorders.

原文English
頁(從 - 到)140-148
頁數9
期刊Trends in Molecular Medicine
17
發行號3
DOIs
出版狀態Published - 2011 3月

All Science Journal Classification (ASJC) codes

  • 分子醫學
  • 分子生物學

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